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Ultragenyx Pharmaceutical Inc. RARE R3KPHARMA

Healthcare · Biotechnology · United States
https://www.ultragenyx.com
Company Profile ↓
$14.88
-48.64% 1Y
Mkt Cap$1.47B
P/E-2.55
P/B-4.90
52W High$36.54
52W Low$14.85
Book Value$-2.96
EPS (TTM)$-5.83

Company Overview

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; Dojolvi for the treatment of long-chain fatty acid oxidation disorders; and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia. The company's products candidates that are in Phase 3 clinical trials include UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; UX111, an AAV9 gene therapy product candidate for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare lysosomal storage disease; DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy for the treatment of patients with ornithine transcarbamylase; and GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome. It also develops UX701, an adeno-associated AAV9 gene therapy which is in Phase 2 clinical trial for the treatment of Wilson liver disease. The company has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; GeneTx; Mereo; University of Pennsylvania; Regeneron; and Abeona. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.

Why Investors Should Care

Steady Revenue Expansion

Revenue has grown at a 190.4% CAGR over the past five years.

Recent Developments

  • Dec 2025 Revenue of $673.00M (+20.2% YoY); net profit $-575.00M.
  • Trailing 12 Months Year-on-year growth — revenue +28.1%.
  • 5-Year Trend Long-term compounding — revenue CAGR 190.4%.

Growth & Price Performance

Compounded Sales Growth

5 Years:190.42%
1 Year:28.10%

Stock Price Performance

1 Year:-48.64%
6 Months:-34.19%
3 Months:-31.08%
1 Month:-42.57%

Highlights auto-generated from reported financials and price data; not investment advice. Verify with official filings before acting.

Technical Snapshot Bearish
52-Week Range 0% of range
$14.85 $36.54
Trend Signals
  • Price vs 50-DMABelow
  • Price vs 200-DMABelow
  • RSI (14)20.62 · Oversold
Price Performance
1M-42.57%
3M-31.08%
6M-34.19%
1Y-48.64%

Auto-generated from price history and fundamentals; not investment advice.

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Price Chart

Robinhood TradingView

Fundamental Growth Charts

Interactive charts for Revenue, Net Income, EBITDA, Free Cash Flow, EPS, ROE, ROCE, Gross/Net/Operating Margin and more — switch metric and period using the dropdowns above. CAGR is computed across the displayed window (quarterly CAGR is annualised). Visual growth charts make it easy to spot multi-year compounding trends at a glance — far faster than reading raw numbers in a table.

PROS

  • Compounding revenue at 190.4% over 5 years.
  • In a confirmed uptrend (50-DMA above 200-DMA).

CONS

  • Trading 59.3% below its 52-week high.
  • RSI at 21 suggests oversold conditions.

* The pros and cons are auto-generated from financial metrics.

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Valuation

Stock P/E
-2.55
Industry PE
24.83
Forward P/E
73.74
PEG Ratio
-
Book Value
$-2.96
Price to Book
-4.90
P/S
2.00
EV/EBITDA
-5.04
Dividend Yield
0.00%

Growth (CAGR)

Revenue 5Y
190.42%
Profit 5Y
-
Revenue (YoY)
28.10%
Earnings (YoY)
-

Profitability & Returns

ROCE
-46.60%
ROE
-
ROA
-24.22%
Profit Margin
-81.73%
Op Margin
-35.05%
Gross Margin
-20.78%
EPS (Latest Qtr)
$-0.90
EPS (TTM)
$-5.83

Balance Sheet & Liquidity

Debt/Equity
-
Quick Ratio
1.41
Current Ratio
1.73
Debt
$1.20B
Total Assets
$1.26B
Current Assets
$657.00M
Working Capital
$567.00M

Ownership

Promoter Holding
3.44%
Chg in Prom Hold
-0.01%
FII / Inst Holding
112.51%
Chg in FII Hold
0.86%

Financial Snapshot

Enterprise Value
$1.47B
Total Revenue (TTM)
$717.00M
EBITDA
$-465.00M
Free Cash Flow
$-221.79M
Operating Cash Flow
$-485.00M
Shares Outstanding
98.59M
Gross Margin
-20.78%
Payout Ratio
0.00%

Peer comparison

Peer companies in the same sector (Healthcare).

#NameCMP $P/E Mkt CapDiv Yld % ROCE %ROE % Rev CAGR 5Y %Profit CAGR 5Y %
1 RARE Ultragenyx Pharmaceutical Inc. R3KPHARMA 14.88 -2.55 $1.47B 0.00% -46.60% - 190.42% -
2 LLY Eli Lilly and Company SPXR3KPHARMA 1,123.91 37.80 $1.00T 0.60% - 102.29% 7.20% 11.41%
3 JNJ Johnson & Johnson SPXR3KPHARMA 269.12 31.22 $648.55B 1.98% 14.88% 25.74% 2.43% 4.76%
4 ABBV AbbVie Inc. SPXR3KPHARMA 248.78 70.48 $439.62B 2.78% 10.47% -129.24% 1.75% -29.06%
5 MRK Merck & Co., Inc. SPXR3KPHARMA 148.46 118.77 $366.28B 2.31% - 6.96% 5.64% 10.01%
6 UNH UnitedHealth Group Incorporated SPXR3KPHARMA 400.84 25.74 $359.79B 2.47% 9.74% 14.15% 10.40% 5.43%
7 NVS Novartis AG PHARMA 137.70 20.80 $261.73B 3.36% 21.09% 30.35% 9.25% 26.22%
8 AZN AstraZeneca PLC NDXPHARMA 160.04 23.99 $248.20B 2.00% 15.97% 21.96% 9.82% 45.96%
9 TMO Thermo Fisher Scientific Inc. SPXR3KPHARMA 603.02 32.49 $222.97B 0.29% 8.14% 13.51% 8.81% 12.95%
Financial Statements Quarterly Results · Profit & Loss · Balance Sheet · Cash Flows · Ratios

Quarterly Results

Figures in USD.

Metric Dec 2015Mar 2016Jun 2016Sep 2016Dec 2016Mar 2017Jun 2017Sep 2017Dec 2017Mar 2018Jun 2018Sep 2018Dec 2018Mar 2019Jun 2019Sep 2019Dec 2019Mar 2020Jun 2020Sep 2020Dec 2020Mar 2021Jun 2021Sep 2021Mar 2022Jun 2022Sep 2022Mar 2023Jun 2023Sep 2023Mar 2024Jun 2024Sep 2024Dec 2024Mar 2025Jun 2025Sep 2025Dec 2025Mar 2026Jun 2026
Revenue -------198.00K2.41M10.68M12.79M11.76M16.26M18.17M24.15M25.80M35.59M36.31M61.71M81.47M91.54M99.39M86.97M81.65M79.94M89.34M90.70M100.50M108.31M98.05M108.83M147.03M139.49M-139.00M166.50M159.93M207.28M136.00M214.00M
Cost of Revenue ----------------------------------29.00M23.00M27.99M29.34M30.00M-
Gross Profit ----------------------------------110.00M143.49M131.94M177.93M106.00M-
Operating Expenses ----------------------------------253.00M251.38M302.83M291.22M275.00M-
Operating Income --53.62M-58.05M-65.78M--69.95M-78.44M-83.71M--96.49M-94.90M-89.65M--99.21M-112.47M-118.03M--120.67M-63.05M-50.31M--106.57M-82.78M-89.83M-136.63M-141.59M-225.07M-154.10M-147.96M-145.10M-165.35M-116.36M-131.99M--143.00M-107.89M-170.89M-113.28M-169.00M-75.00M
EBITDA -----------------------------------127.00M-91.35M-156.79M-99.43M-155.00M-
Interest Expense ----------------------------------14.00M14.04M14.15M19.47M21.00M-
Pretax Income -----------------------------------150.00M-114.00M-179.54M-127.69M-184.00M-
Tax Provision ----------------------------------1.00M947.00K873.00K870.00K1.00M-
Net Income -55.22M-52.76M-56.92M-64.91M-71.29M-68.29M-72.89M-79.23M-81.73M30.25M-52.73M-87.31M-87.83M-96.76M-99.17M-112.99M-93.81M-119.03M25.32M-68.84M-24.01M-136.14M-122.43M-73.00M-152.32M-158.16M-245.11M-163.97M-159.83M-159.65M-170.68M-131.60M-133.52M--151.00M-114.95M-180.41M-128.56M-185.00M-92.00M
Diluted EPS ------1.63---0.62-1.06-1.74-1.73-1.82-1.72---2.050.41-1.13-0.37--1.81-1.08-2.19-2.26-3.50-2.33-2.25-2.23-2.03-1.52-1.40--1.57-1.17-1.81-1.29-1.84-0.90
R&D Expense -40.41M43.33M48.71M-51.27M58.44M60.41M-75.50M76.83M70.04M-78.11M96.05M100.14M-112.96M80.71M87.31M-147.52M113.20M113.42M143.16M154.53M237.30M165.70M164.95M157.25M178.49M161.50M170.11M-166.00M164.74M216.21M-187.00M167.00M

Profit & Loss (Annual)

Figures in USD.

Metric Dec 2012Dec 2013Dec 2014Dec 2015Dec 2016Dec 2017Dec 2018Dec 2019Dec 2020Dec 2021Dec 2022Dec 2023Dec 2024Dec 2025
Revenue ----133.00K2.61M51.49M103.71M271.03M-363.33M434.00M560.00M673.00M
Cost of Revenue ----------28.32M45.00M77.00M109.00M
Gross Profit ----------335.01M389.00M483.00M564.00M
Operating Expenses ----------983.93M958.00M1.02B1.10B
Operating Income -15.98M-32.28M-56.78M-147.74M-248.01M-328.94M-371.37M-424.17M-330.12M--648.92M-569.00M-536.00M-535.00M
EBITDA -----------640.49M-517.00M-468.00M-474.00M
Interest Expense ----------43.02M66.00M63.00M62.00M
Pretax Income -----------701.73M-609.00M-567.00M-571.00M
Tax Provision ----------5.70M-2.00M2.00M4.00M
Net Income -16.33M-35.07M-59.80M-145.62M-245.87M-302.14M-197.61M-402.73M-186.57M--707.42M-607.00M-569.00M-575.00M
Diluted EPS ---------3.07--10.12-8.25-6.29-5.83
R&D Expense 12.64M27.83M45.97M114.74M183.20M231.64M294.00M357.36M412.08M497.15M705.79M648.00M698.00M750.00M

Balance Sheet (Annual)

Figures in USD.

Metric Dec 2011Dec 2012Dec 2013Dec 2014Dec 2015Dec 2016Dec 2017Dec 2018Dec 2019Dec 2020Dec 2021Dec 2022Dec 2023Dec 2024Dec 2025
Total Assets --59.65M197.97M559.57M540.63M490.75M719.56M1.14B1.76B-1.55B1.49B1.50B1.53B
Current Assets -----------883.90M732.18M817.00M951.00M
Cash & Equivalents 10.64M86.19M7.43M24.32M93.57M161.12M100.49M113.43M433.58M713.53M-132.94M213.58M174.00M421.00M
Inventory -----------26.77M33.97M45.00M52.00M
Receivables -----------40.45M73.39M122.00M158.00M
Total Liabilities --9.54M13.02M28.48M66.65M107.30M110.65M481.73M605.18M-1.19B1.22B1.24B1.60B
Current Liabilities --5.92M12.52M27.92M60.26M71.01M74.72M103.30M189.61M-261.21M280.44M344.00M384.00M
Long Term Debt ---------------
Total Debt -----------31.59M43.17M40.00M36.00M
Total Equity -7.96M-27.05M-74.82M184.94M531.09M473.97M383.45M608.91M653.76M1.15B-352.49M275.41M255.00M-80.00M
Shares Outstanding -----------70.20M82.33M92.55M96.65M

Cash Flows (Annual)

Figures in USD.

Metric Dec 2012Dec 2013Dec 2014Dec 2015Dec 2016Dec 2017Dec 2018Dec 2019Dec 2020Dec 2021Dec 2022Dec 2023Dec 2024Dec 2025
Operating Cash Flow -----160.97M-253.84M-290.57M-345.38M-132.22M--380.46M-475.00M-414.00M-466.00M
Investing Cash Flow ----91.12M55.48M-33.33M-13.04M-179.12M--291.65M168.00M-18.00M236.00M
Financing Cash Flow ----138.68M136.27M336.85M679.31M600.27M-501.21M388.00M399.00M478.00M
Capital Expenditure -1.09M-407.00K-2.15M-4.96M-10.19M-2.79M-4.08M-24.83M-43.91M--146.12M-47.00M-20.00M-21.00M
Free Cash Flow -----171.16M-256.64M-294.64M-370.21M-176.12M--526.59M-522.00M-434.00M-487.00M
Net Change in Cash -----------170.91M81.00M-33.00M248.00M

Ratios (Annual)

Figures in %.

Metric Dec 2012Dec 2013Dec 2014Dec 2015Dec 2016Dec 2017Dec 2018Dec 2019Dec 2020Dec 2021Dec 2022Dec 2023Dec 2024Dec 2025
Gross Margin % ----------92.2%89.6%86.2%83.8%
Operating Margin % -----186,471.4%-12,593.5%-721.2%-409.0%-121.8%--178.6%-131.1%-95.7%-79.5%
Net Margin % -----184,867.7%-11,567.3%-383.7%-388.3%-68.8%--194.7%-139.9%-101.6%-85.4%
ROE % 60.4%46.9%-32.3%-27.4%-51.9%-78.8%-32.5%-61.6%-16.2%--200.7%-220.4%-223.1%718.8%
ROCE % --60.1%-30.6%-27.8%-51.6%-78.4%-57.6%-41.1%-21.0%--50.5%-47.0%-46.2%-46.6%

Shareholding Pattern

Insiders
3.44%
Institutions
112.51%
Public Float
116.52%

Top Institutional Holders

#Holder% HeldSharesValue
1 Blackrock Inc. 10.81% 10.65M $154.59M
2 RTW Investments LP 7.91% 7.80M $113.13M
3 Vanguard Portfolio Management LLC 6.89% 6.79M $98.52M
4 AQR Capital Management, LLC 6.65% 6.56M $95.20M
5 State Street Corporation 6.25% 6.16M $89.36M
6 Vanguard Capital Management LLC 4.36% 4.30M $62.34M
7 Two Sigma Investments, LP 3.67% 3.62M $52.54M
8 Sands Capital Management, LLC 3.20% 3.16M $45.83M
9 Jacobs Levy Equity Management, Inc. 2.98% 2.94M $42.66M
10 Frontier Capital Management Company LLC 2.89% 2.85M $41.32M

Analyst View

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Latest News

Recent headlines for RARE

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Pharvaris’ stock jumps on pivotal success in rare genetic disease - Endpoints News

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Google News Sat, 05 Sep 2026

Ultragenyx (RARE) Stock Looks Fully Valued Following Its Phase 3 Setback - finance.yahoo.com

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RARE — Frequently Asked Questions

What is the current share price of Ultragenyx Pharmaceutical Inc. (RARE)?

As of 2026-09-19 21:16 PDT, Ultragenyx Pharmaceutical Inc. (RARE) trades at $14.88 on NasdaqGS. Its 52-week range is $14.85 to $36.54.

What is the market capitalisation of RARE?

Ultragenyx Pharmaceutical Inc. (RARE) has a market capitalisation of $1.47B on NasdaqGS.

What is the P/E ratio of RARE?

RARE trades at a trailing price-to-earnings (P/E) ratio of -2.55. The industry average P/E is 24.83. Its price-to-book (P/B) ratio is -4.90.

Is RARE a good stock to buy?

This page provides a data-driven analysis of Ultragenyx Pharmaceutical Inc. (RARE), including valuation ratios, profitability, growth trends, auto-generated pros and cons, peer comparison and AI exposure. Review these fundamentals alongside your own research and risk tolerance. This is informational content, not investment advice.

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